US

United States

US

North America

Regulatory Overview

United States is regulated by the FDA (Food and Drug Administration). The country has 2 documented regulatory pathways for accessing unapproved or unlicensed medicines, covering named patient programs, compassionate use schemes, and special access arrangements.

Regulatory Authority

FDA (Food and Drug Administration)

Regulatory Pathways (2)

Emergency Investigational New Drug application for life-threatening situations requiring immediate treatment where there is no time to submit a formal IND. FDA may authorize treatment by telephone.

Application Form: Emergency IND Request (telephone/fax)
Legal Basis: 21 CFR 312.310
Eligibility: Serious or life-threatening condition with no locally registered alternative
Reference: FDA Emergency IND Guidance

FDA program allowing patients with serious or immediately life-threatening conditions to access investigational drugs, biologics, or medical devices outside of clinical trials.

Application Form: FDA Form 3926 (Individual Patient IND)
Legal Basis: 21 CFR Part 312 Subpart I
Eligibility: Serious or life-threatening condition with no locally registered alternative
Reference: FDA Expanded Access Guidance

Drugs Available (266)

DrugAccess RouteStatusLead Time
alglucosidase alfa
cytarabine
eliglustat
vitamin A (retinol)
furosemide
eribulin mesylate
Digoxin Immune Fab
Sucralfate
Dorzolamide
Pravastatin
Temsirolimus
naproxen
Memantine
propranolol
Berotralstat
Everolimus
Ursodeoxycholic acid
tamoxifen citrate
atropine sulfate
metoprolol
carbamazepine
ruxolitinib
Corticosteroids
Melphalan
mycophenolate mofetil
Epoprostenol
Fludarabine
Sodium Phenylbutyrate
etanercept
Denosumab
dapsone
Tofacitinib
mavacamten
diethylcarbamazine
mannose
minoxidil
ibuprofen
gabapentin
Hydroxychloroquine
cyclophosphamide
Anakinra
bosentan
bimatoprost
testosterone
Artificial tears
Rho(D) immune globulin
Baclofen
Dexrazoxane
Vigabatrin
Clobazam
triheptanoin
Beremagene geperpavec
C1 esterase inhibitor (human)
phacoemulsification surgery
fosdenopterin
melatonin
amiodarone
Total Parenteral Nutrition
Growth hormone (somatropin)
ascorbic acid
hormone replacement therapy (estrogen/progesterone)
Busulfan
Digoxin
Bimekizumab-bkzx
Interferon-gamma
Vitamin B12 (Cobalamin)
Romidepsin
Interferon gamma-1b
collagenase clostridium histolyticum
Crizotinib
physical therapy and orthotic management
Retinoids (Tretinoin)
Branched-Chain Amino Acids
eculizumab
miglustat
GM-CSF
Palovarotene
Botulism Antitoxin Heptavalent (A, B, C, D, E, F, G) - (Equine)
lonafarnib
Betaine anhydrous
tizanidine
Octreotide
aspirin
oxybutynin
Valproic Acid
tocilizumab
sclerostin antibody
Immunoglobulin
Mesalamine
alpha-L-iduronidase
L-DOPA
Intravenous Immunoglobulin (IVIG)
Phenytoin
Bisphosphonates (pamidronate)
Metronidazole
Nitisinone
Human Chorionic Gonadotropin (hCG)
Lomitapide
treprostinil
sirolimus
temozolomide
Carnitine
brivaracetam
Pembrolizumab
Infliximab
folic acid
Pyridoxine
Acetazolamide
Cibinqo
Somatrogon-ghla
Clomiphene Citrate
Angiotensin-Converting Enzyme Inhibitors (Enalapril)
Multivitamin with Calcium and Vitamin D
Lorenzo's oil
levothyroxine sodium
ivermectin
botulinum toxin type A
Levothyroxine
mexiletine
Crizanlizumab
azathioprine
sunscreen (broad spectrum SPF 50+)
Somatropin
Mifepristone
galantamine
ambrisentan
Deflazacort
Brentuximab vedotin
L-Methylfolate
Dabrafenib
L-carnitine
insulin
luspatercept
trabectedin
prednisolone
vosoritide
Arsenic trioxide
cysteamine bitartrate
Edaravone
Tegaserod
Vorasidenib
rituximab
Dapagliflozin
allopurinol
Carboplatin
Topical corticosteroids
Trametinib
Ivosidenib
estradiol
Erythromycin
Erythropoietin
Doxycycline
calcium carbonate
coenzyme Q10
Patisiran
Tolvaptan
cerliponase alfa
belumosudil
Sodium Thiosulfate
adalimumab
Imiglucerase
Amphotericin B liposomal
ranibizumab
Midostaurin
metformin
Lumasiran
Methotrexate
Nitazoxanide
carglumic acid
Alendronate Sodium
calcitriol
doxorubicin
Pyridostigmine
Levetiracetam
Cenegermin
Acitretin
prednisone
Hydroxyurea
salicylic acid
Insulin glargine
cochlear implant
Pyridostigmine Bromide
aflibercept
Ravulizumab
laronidase
Corticotropin
Romiplostim
Bleomycin
hydroxycobalamin
Pyrimethamine
Metreleptin
Laser photocoagulation therapy
Sulfadiazine
acetaminophen
pregabalin
riboflavin
N-acetylcysteine
NovoSeven
Occupational and Physical Therapy Support
eptinezumab
Pamidronate
nitroglycerin
Sacrosidase
Mipomersen
ezetimibe
Belzutifan
atorvastatin
bevacizumab
proton pump inhibitors (omeprazole)
Ketoconazole
mecasermin
celecoxib
Biotin
Imiquimod
cyclosporine ophthalmic
Growth Hormone
Gonadotropin
deferasirox
Diatrizoate
Tafamidis
urea
Levodopa/Carbidopa
Lanreotide
Olipudase alfa
cyanocobalamin
Eteplirsen
Sodium Bicarbonate
diazoxide
baricitinib
Azacitidine
Cystagon
Zoledronic Acid
Hypertonic saline
copper histidinate
Clonazepam
Zidovudine
emollients with keratolytics
warfarin
Vitamin K1 (Phytonadione)
cornstarch
disopyramide
asciminib
Vinblastine
selumetinib
Sunitinib
mannose-1-phosphate
Cisplatin
Calcipotriol
Imatinib
imatinib mesylate
nimodipine
Colchicine
Follitropin alfa
surgical repair
Tamoxifen
Tacrolimus
Artificial tears (Hypromellose)
Ribavirin
vitamin D3
Sildenafil
sotalol
Hematopoietic stem cell transplantation
Tretinoin
Diazepam
Timolol maleate
Amnioreduction
US
United States
ISO CodeUS
RegionNorth America
Regulatory AuthorityFDA (Food and Drug Administration)
Pathways2 documented
Drugs Available266 drug(s)